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Alex Yiu was born a seemingly healthy baby. But by age 2, his muscle control and speech were deteriorating. His baffling condition took a decade to diagnose. The reanalysis of a DNA test was the key.
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President Donald Trump wants to streamline the U.S. Food and Drug Administration so people with rare diseases can get medication faster. But one Florida...
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Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives by doing so. But nearly a third of the medicines aren't new or were repurposed many times for financial gain.
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You often hear about children with rare afflictions that can’t be helped. Here are some young people who beat the odds:A 2-year-old with Shone's syndrome,…